PB0630 - Approach to prophylaxis in young boys with severe Haemophilia A in the era of emicizumab: a single centre experience

Monday, June 24, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Presenting Author(s)

SB

Saket Badle

Consultant Paediatric Haematologist
Paediatric Haemostasis Unit, Oxford Haemophilia and Thrombosis Centre
Oxford, England, United Kingdom

Co-Author(s)

Marie C. Eales, BA (Hons) MSc ANP

Haemophilia Advanced Nurse Practitioner
Paediatric Haemostasis Unit, Oxford Haemophilia and Thrombosis Centre
Oxford, England, United Kingdom
OP

Omer Pervaiz

Haematology Registrar
Paediatric Haemostasis Unit, Oxford Haemophilia and Thrombosis Centre
Oxford, England, United Kingdom
AW

Alice Wilkinson

Haemophilia Specialist Nurse
Paediatric Haemostasis Unit, Oxford Haemophilia and Thrombosis Centre
Oxford, England, United Kingdom
SP

Sarah Pool

Haemophilia Specialist Nurse
Paediatric Haemostasis Unit, Oxford Haemophilia and Thrombosis Centre
Oxford, England, United Kingdom
Ay

Ashley yburan

Haemophilia Research Nurse
Paediatric Haemostasis Unit, Oxford Haemophilia and Thrombosis Centre
Oxford, England, United Kingdom
LG

Lisa Gueran

Haemophilia Specialist Physiotherapist
Paediatric Haemostasis Unit, Oxford Haemophilia and Thrombosis Centre
Oxford, England, United Kingdom
GH

Georgina Hall

Consultant Paediatric Haematologist
Paediatric Haemostasis Unit, Oxford Haemophilia and Thrombosis Centre
Oxford, England, United Kingdom
NB

Neha Bhatnagar

Consultant Paediatric Haematologist
Paediatric Haemostasis Unit, Oxford Haemophilia and Thrombosis Centre
Oxford, England, United Kingdom

Background: Regular replacement (prophylaxis) is standard-of-care in severe Haemophilia to maintain haemostasis, prevent joint-haemorrhage and arthropathy. Starting prophylaxis at a young age has best joint outcomes and reduction in incidence of intracranial haemorrhage in young-children. UK practice in severe haemophilia A(SHA) is to provide lifelong-prophylaxis with either recombinant Factor VIII(rFVIII) or non-factor-replacement i.e. emicizumab.

Aims: We describe our experience of prophylaxis with emicizumab in boys with SHA.

Methods: Data obtained from local database and clinical-notes of boys starting emicizumab-prophylaxis between June 2019-June 2023. Patient demographics, FVIII exposure, inhibitor and bleed data is presented in Table 1, prophylaxis discussion with reasons for starting emicizumab is described.

Results: 3(18.7%) patients started emicizumab due to ITI to allow safe prophylaxis, the rest (>80%) started Emicizumab due to the other factors- problematic venous access, needle phobia, limited access to clinical services (COVID-19, travel to centre, parental work commitments). Parental preference to start/ switch to emicizumab was due to similar factors.

We offer choice of emicizumab or rFVIII-prophylaxis at initial prophylaxis consultation. Emicizumab is also offered to patients on established rFVIII-prophylaxis with persistent venous-access issues or noncompliance and is part of Immune-tolerance-Induction (ITI) in patients with FVIII-inhibitor. We discuss the longer-term aim to introduce rFVIII-prophylaxis when older or peripheral-access permits, in all patients on emicizumab.

Conclusion(s): Emicizumab was offered as a bridge to starting rFVIII prophylaxis in boys with FVIII-inhibitor, difficult peripheral venous access or non-compliance. Emicizumab offers good bleed protection, in our cohort on emicizumab there was minimal need for additional treatment. Prophylaxis can start at a much younger age (with supporting evidence from HAVEN 7 trial data) providing protection from early joint as well as other-site bleeds. We continue to advocate the use of rFVIII as the gold standard, but emicizumab has revolutionised the management of SHA and rFVIII prophylaxis training can start in a more controlled fashion.